Welcome to Gene Therapy Net
Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.
Gene-Therapy Company Crispr Drops as FDA Puts Trial on Hold
Posted on: 10 June 2018, source: Bloomberg
Crispr Therapeutics AG plunged after the gene-editing company said a planned trial of its treatment to help people with sickle-cell disease was being put on hold by U.S. regulators. Crispr is developing the therapy with Vertex Pharmaceuticals Inc., and the trial had yet to begin. The companies said in a statement Wednesday that the Food and Drug Administration placed a halt on the experiment until the agency could get more information from the companies.
Crispr Therapeutics AG plunged after the gene-editing company said a planned trial of its treatment to help people with sickle-cell disease was being put on hold by U.S. regulators. Crispr is developing the therapy with Vertex Pharmaceuticals Inc., and the trial had yet to begin. The companies said in a statement Wednesday that the Food and Drug Administration placed a halt on the experiment until the agency could get more information from the companies.
A Harvard startup is using gene therapy to reverse ageing in dogs, humans are next
Posted on: 14 May 2018, source: Moneycontrol.com
In order to achieve the objective to “have the body and mind of a 22-year-old but the experience of a 130-year-old,” a Harvard professor backed startup is planning to rejuvenate dogs using gene therapy. And, if results are positive, the same approach could be used in humans. Rejuvenate Bio, co-founded by George Church of Harvard Medical School, has already carried out age-defying tests on beagles and claims it will make animals “younger" by adding new DNA instructions to their bodies.
In order to achieve the objective to “have the body and mind of a 22-year-old but the experience of a 130-year-old,” a Harvard professor backed startup is planning to rejuvenate dogs using gene therapy. And, if results are positive, the same approach could be used in humans. Rejuvenate Bio, co-founded by George Church of Harvard Medical School, has already carried out age-defying tests on beagles and claims it will make animals “younger" by adding new DNA instructions to their bodies.
Latest Up and Coming Gene Therapies
Posted on: 8 May 2018, source: GEN
Gene therapy’s numbers look promising. In a year full of biopharma mergers and acquisitions, one of the biggest deals has been Novartis’ planned $8.7 billion purchase of AveXis, whose lead candidate AVXS-101 has advanced into a pivotal trial following positive Phase I data published in November in The New England Journal of Medicine. Additional figures furnished to GEN by Informa show the extent of gene-therapy development: Informa Pharma Intelligence’s Trialtrove database records 729 gene therapies as having been developed, of which nearly two-thirds (461) were preclinical. Those therapies have been assessed in 1,855 clinical trials, most in early phases: 657 in Phase I, 509 in Phase I/II, and 455 in Phase II. As for later development, 89 have reached Phase III, 32 are in Phase II/III, and 28 in Phase IV.
Gene therapy’s numbers look promising. In a year full of biopharma mergers and acquisitions, one of the biggest deals has been Novartis’ planned $8.7 billion purchase of AveXis, whose lead candidate AVXS-101 has advanced into a pivotal trial following positive Phase I data published in November in The New England Journal of Medicine. Additional figures furnished to GEN by Informa show the extent of gene-therapy development: Informa Pharma Intelligence’s Trialtrove database records 729 gene therapies as having been developed, of which nearly two-thirds (461) were preclinical. Those therapies have been assessed in 1,855 clinical trials, most in early phases: 657 in Phase I, 509 in Phase I/II, and 455 in Phase II. As for later development, 89 have reached Phase III, 32 are in Phase II/III, and 28 in Phase IV.
Gene therapy for beta-thalassemia safe, effective in people
Posted on: 19 April 2018, source: EurekAlert
In a powerful example of bench-to-bedside science showing how observations made in the lab can spark life-altering therapies in clinic, an international team of clinician-investigators has announced that gene therapy for patients with a severe form of the blood disorder beta-thalassemia can be safe and effective. Led by study director Philippe Leboulch, MD, a sponsored collaborator in Brigham and Women's Hospital's Division of Genetics and lecturer in medicine at Harvard Medical School, the research team reports that a one-time treatment with the gene therapy known as LentiGlobin BB305 vector reduced or eliminated the need for blood transfusions in 22 patients with severe beta-thalassemia. The team's results are published in the April 19 issue of The New England Journal of Medicine.
In a powerful example of bench-to-bedside science showing how observations made in the lab can spark life-altering therapies in clinic, an international team of clinician-investigators has announced that gene therapy for patients with a severe form of the blood disorder beta-thalassemia can be safe and effective. Led by study director Philippe Leboulch, MD, a sponsored collaborator in Brigham and Women's Hospital's Division of Genetics and lecturer in medicine at Harvard Medical School, the research team reports that a one-time treatment with the gene therapy known as LentiGlobin BB305 vector reduced or eliminated the need for blood transfusions in 22 patients with severe beta-thalassemia. The team's results are published in the April 19 issue of The New England Journal of Medicine.
13-Year-Old Boy Is First Person in US to Receive Newly Approved Gene Therapy for Blindness
Posted on: 22 March 2018, source: Gizmodo
On Tuesday, a 13-year-old boy from New Jersey was at the center of medical history as he became the first person in the US to receive an FDA-approved gene therapy for an inherited disease. The event marks the beginning of a new era of medicine, one in which devastating genetic conditions that we are born with can be simply edited out of our DNA with the help of modern biomedical technologies. The therapy, Luxturna, from Spark Therepeutics, was approved by the FDA in December to treat a rare, inherited form of blindness. Its price tag, set at $850,000—or $425,000 per eye—made it the most expensive drug in the US and sparked mass sticker-shock. But the therapy, which in high-profile clinical trials has allowed patients to see the stars for the first times, also offered the almost miraculous possibility of giving sight to the blind.
On Tuesday, a 13-year-old boy from New Jersey was at the center of medical history as he became the first person in the US to receive an FDA-approved gene therapy for an inherited disease. The event marks the beginning of a new era of medicine, one in which devastating genetic conditions that we are born with can be simply edited out of our DNA with the help of modern biomedical technologies. The therapy, Luxturna, from Spark Therepeutics, was approved by the FDA in December to treat a rare, inherited form of blindness. Its price tag, set at $850,000—or $425,000 per eye—made it the most expensive drug in the US and sparked mass sticker-shock. But the therapy, which in high-profile clinical trials has allowed patients to see the stars for the first times, also offered the almost miraculous possibility of giving sight to the blind.
Gene therapy found safe in trial of Pompe disease treatment
Posted on: 22 February 2018, source: UPI news
The first human trial of gene therapy to treat respiratory problems in early-onset Pompe disease of children was found safe, according to health researchers in Florida. University of Florida Health scientists conducted a successful trial of nine participants who had the incurable disease, which damages muscle and nerve cells throughout the body. Results of the treatment, which utilizes an adeno-associated virus, were published in the journal Human Gene Therapy Clinical Development.
The first human trial of gene therapy to treat respiratory problems in early-onset Pompe disease of children was found safe, according to health researchers in Florida. University of Florida Health scientists conducted a successful trial of nine participants who had the incurable disease, which damages muscle and nerve cells throughout the body. Results of the treatment, which utilizes an adeno-associated virus, were published in the journal Human Gene Therapy Clinical Development.
High-dose AAV gene therapy appears toxic in monkeys and piglets
Posted on: 6 February 2018, source: Science
A gene therapy trial that recently led to dramatic benefits for babies born with a fatal neuromuscular condition has raised hopes for using a similar approach to treat other diseases. But a new animal study suggests that the high doses (2x10E14 genome copies per kilogram body weight) of gene-carrying viruses used in such treatments may not always be as safe as the human clinical trial indicated. In the new research, the disclosure of which briefly sent the stock prices of several gene therapy companies plummeting yesterday, researchers injected a handful of young monkeys and pigs with many copies of adeno-associated virus 9 (AAV9), a normally harmless virus that infects neurons and is increasingly being used to ferry therapeutic genes into cells to treat neuromuscular diseases. Within days, some of the animals developed severe liver and neuron damage.
A gene therapy trial that recently led to dramatic benefits for babies born with a fatal neuromuscular condition has raised hopes for using a similar approach to treat other diseases. But a new animal study suggests that the high doses (2x10E14 genome copies per kilogram body weight) of gene-carrying viruses used in such treatments may not always be as safe as the human clinical trial indicated. In the new research, the disclosure of which briefly sent the stock prices of several gene therapy companies plummeting yesterday, researchers injected a handful of young monkeys and pigs with many copies of adeno-associated virus 9 (AAV9), a normally harmless virus that infects neurons and is increasingly being used to ferry therapeutic genes into cells to treat neuromuscular diseases. Within days, some of the animals developed severe liver and neuron damage.
Gene Therapy News
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Mon, 25 Jun 2018 12:49 GMT - 'Home run' gene therapy for Duchenne muscular dystrophy: Investor-oriented coverage pitches promise but …
Mon, 25 Jun 2018 15:24 GMT - Gene therapy cures rat's paralysis—could human beings be next?
Mon, 25 Jun 2018 11:05 GMT - Medical Discovery News: Gene therapy provides hope for rheumatoid arthritis sufferers
Mon, 25 Jun 2018 12:00 GMT - Early results for gene therapy for muscular dystrophy raises hope
Sat, 23 Jun 2018 00:40 GMT
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Recent early view article in Journal of Gene Medicine:
You can buy a unique research paper online on gene therapy topics here.